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salanersen
- Development stage
- Phase 3
- Lead developer
- Biogen
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Injection
01
Overview
Salanersen is an investigational antisense oligonucleotide (ASO) designed to increase the production of survival motor neuron (SMN) protein by modulating the splicing of the SMN2 gene. It is being developed for the treatment of spinal muscular atrophy (SMA), a rare neuromuscular disorder caused by mutations in the SMN1 gene that result in insufficient levels of functional SMN protein. Salanersen acts as an SMN2 stimulant, promoting increased expression of full-length SMN protein from the backup SMN2 gene. The drug is administered via injection and has orphan drug designation in the United States[1][3][4]. Its mechanism and therapeutic rationale are similar to those of nusinersen but it is a distinct molecule.
- Other names
- salanersen sodiumall-P-ambo-(5'?3')-[2?-O-(2-Methylamino)-2-oxoethyl]-P-thiocytidylyl-(5'?3')-[2?-O-(2-methylamino)-2-oxoethyl]adenylyl
02
Targets
SMN2 (Survival motor neuron 2)
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